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Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial.
Heijerman, Harry G M; McKone, Edward F; Downey, Damian G; Van Braeckel, Eva; Rowe, Steven M; Tullis, Elizabeth; Mall, Marcus A; Welter, John J; Ramsey, Bonnie W; McKee, Charlotte M; Marigowda, Gautham; Moskowitz, Samuel M; Waltz, David; Sosnay, Patrick R; Simard, Christopher; Ahluwalia, Neil; Xuan, Fengjuan; Zhang, Yaohua; Taylor-Cousar, Jennifer L; McCoy, Karen S.
Afiliación
  • Heijerman HGM; Department of Pulmonology, University Medical Center Utrecht, Utrecht, Netherlands. Electronic address: h.g.m.heijerman@umcutrecht.nl.
  • McKone EF; St Vincent's University Hospital and University College Dublin School of Medicine, Dublin, Ireland.
  • Downey DG; Centre for Experimental Medicine, Queen's University Belfast, Belfast, UK.
  • Van Braeckel E; Department of Respiratory Medicine, Ghent University Hospital, Ghent, Belgium.
  • Rowe SM; Division of Pulmonary, Allergy, and Critical Care Medicine, University of Alabama at Birmingham, Birmingham, AL, USA.
  • Tullis E; Division of Respirology, St Michael's Hospital, University of Toronto, Toronto, ON, Canada.
  • Mall MA; Department of Pulmonology, Immunology and Intensive Care Medicine, Charité-Universitätsmedizin Berlin, Berlin, Germany; Berlin Institute of Health, Berlin, Germany; German Center for Lung Research, Berlin, Germany.
  • Welter JJ; Division of Pediatric Pulmonology, Allergy, Immunology, and Sleep Medicine, New York Medical College, Valhalla, NY, USA.
  • Ramsey BW; Department of Pediatrics, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA, USA.
  • McKee CM; Vertex Pharmaceuticals, Boston, MA, USA.
  • Marigowda G; Vertex Pharmaceuticals, Boston, MA, USA.
  • Moskowitz SM; Vertex Pharmaceuticals, Boston, MA, USA.
  • Waltz D; Vertex Pharmaceuticals, Boston, MA, USA.
  • Sosnay PR; Vertex Pharmaceuticals, Boston, MA, USA.
  • Simard C; Vertex Pharmaceuticals, Boston, MA, USA.
  • Ahluwalia N; Vertex Pharmaceuticals, Boston, MA, USA.
  • Xuan F; Vertex Pharmaceuticals, Boston, MA, USA.
  • Zhang Y; Vertex Pharmaceuticals, Boston, MA, USA.
  • Taylor-Cousar JL; Department of Medicine and Pediatrics, National Jewish Health, Denver, CO, USA.
  • McCoy KS; Department of Pediatrics, Nationwide Children's Hospital, Ohio State University, Columbus, OH, USA.
Lancet ; 394(10212): 1940-1948, 2019 11 23.
Article en En | MEDLINE | ID: mdl-31679946
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Pirazoles / Piridinas / Pirrolidinas / Quinolonas / Regulador de Conductancia de Transmembrana de Fibrosis Quística / Fibrosis Quística / Benzodioxoles / Agonistas de los Canales de Cloruro / Aminofenoles / Indoles Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Adolescent / Child / Female / Humans / Male Idioma: En Revista: Lancet Año: 2019 Tipo del documento: Article
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Pirazoles / Piridinas / Pirrolidinas / Quinolonas / Regulador de Conductancia de Transmembrana de Fibrosis Quística / Fibrosis Quística / Benzodioxoles / Agonistas de los Canales de Cloruro / Aminofenoles / Indoles Tipo de estudio: Clinical_trials / Prognostic_studies Límite: Adolescent / Child / Female / Humans / Male Idioma: En Revista: Lancet Año: 2019 Tipo del documento: Article